Huntington’s disease treatment milestone: uniQure secures priority FDA review for one-time gene therapy
uniQure's Huntington's gene therapy AMT-130 secures FDA priority review, with a May 1, 2027 decision target. Explore the clinical evidence.
Pharma & Biotech
Pharma & Biotech
AMT-130 slowed Huntington’s progression by 75%. uniQure has now filed for FDA approval
uniQure has submitted AMT-130 for accelerated U.S. approval after three-year Phase 1/2 data showed a 75% slowing of Huntington’s disease progression against a matched natural-history control.
Pharma & Biotech
Can uniQure turn the FDA’s AMT-130 reversal into a Huntington’s disease approval?
The Food and Drug Administration will accept uniQure’s three-year AMT-130 data as the primary basis for an accelerated approval filing, but external controls, irreversible brain delivery and confirmatory evidence remain decisive risks.
Pharma & Biotech
FDA accepts uniQure’s AMT-130 filing strategy after months of regulatory uncertainty
Explore why the FDA reopened uniQure’s AMT-130 approval path and what evidence, surgical and confirmatory trial risks remain before review.